Ongoing Clinical Trials for Efzofitimod (ATYR1923)
ATYR1923-C-004: EFFICACY AND SAFETY OF INTRAVENOUS EFZOFITIMOD IN PATIENTS WITH PULMONARY SARCOIDOSIS
We are conducting a Phase 3 clinical trial investigating the efficacy and safety of efzofitimod (ATYR1923) in patients with pulmonary sarcoidosis.
Pulmonary sarcoidosis is an inflammatory disease of unknown cause, characterized by the formation of granulomas, clumps of inflammatory cells, in the lungs. If left untreated, this inflammation can lead to scarring (fibrosis) of the lung tissue resulting in permanent lung damage and in some cases death. Efzofitimod works by dampening immune responses in inflammatory disease states, with the goal of resolving inflammation and preventing progression to fibrosis. Efzofitimod’s mechanism of action overlaps with the cellular pathology observed in pulmonary sarcoidosis.
The EFZO-FIT™ study is a global Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous (IV) efzofitimod 3mg/kg and 5 mg/kg versus placebo in patients with symptomatic pulmonary sarcoidosis. This will be a 52 week study with patients receiving either efzofitimod or placebo once a month for a total of 12 doses. The study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid, with or without immunosuppressant therapy.
The study intends to enroll 264 patients at centers throughout North America, Europe and Japan. The study will incorporate a forced steroid taper. The purpose of this study is to evaluate whether efzofitimod treatment can reduce the dose of oral corticosteroid compared to placebo. The study will also evaluate if efzofitimod will maintain lung function and symptom control when steroids are reduced.
For more information, please visit clinicaltrials.gov (NCT05415137).
Completed Clinical Trials for Efzofitimod (ATYR1923)
ATYR1923-C-002: Investigational Trial of ATYR1923 for the Treatment of Pulmonary Sarcoidosis
In September 2021, we announced positive results from a Phase 1b/2a clinical trial evaluating efzofitimod (ATYR1923) in patients with pulmonary sarcoidosis.
The Phase 1b/2a trial was a multiple-ascending dose, double-blind, placebo-controlled study evaluating efzofitimod in 37 patients with pulmonary sarcoidosis. The purpose of this study was to determine if efzofitimod was safe and tolerable in patients with pulmonary sarcoidosis who underwent a protocol-guided oral corticosteroid taper. This study was also designed to explore whether efzofitimod had the potential to reduce oral corticosteroid use, as well as the effects efzofitimod may have had on pulmonary sarcoidosis by measures of lung function, health-related quality of life assessments and disease biomarkers.
aTyr collaborated with the Foundation for Sarcoidosis Research (FSR), the nation’s leading nonprofit organization dedicated to finding a cure for sarcoidosis and improving care for sarcoidosis patients.
clinicaltrials.gov
Data Release
ATYR1923-C-003: Investigational Trial of ATYR1923 for the Treatment of COVID-19 Related Severe Respiratory Complications
In the first quarter of 2021 we announced positive data from our Phase 2 clinical trial evaluating efzofitimod in patients with COVID-19 related severe respiratory complications. The trial was a randomized, double-blind, placebo-controlled study designed to investigate the safety and preliminary efficacy of efzofitimod as compared to placebo in 32 hospitalized COVID-19 patients with severe respiratory complications who did not require mechanical ventilation. Patients enrolled in the trial were randomized 1:1:1 to receive a single intravenous dose of either 1.0 or 3.0 mg/kg efzofitimod or placebo. Patients were followed for 60 days post treatment.
clinicaltrials.gov
Data Release
ACTRN12617001446358: EVALUATION OF THE SAFETY, TOLERABILITY, IMMUNOGENICITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF INTRAVENOUS ATYR1923 IN HEALTHY VOLUNTEERS
In the second quarter of 2018 we announced positive data from our Phase 1 clinical trial of efzofitimod. This first-in-human, randomized, double-blind, placebo-controlled study was designed to investigate the safety, tolerability, immunogenicity and pharmacokinetics of intravenous efzofitimod in healthy volunteers. The Phase 1 study enrolled 36 healthy volunteers who were randomized to one of six cohorts and received a single infusion of intravenous efzofitimod or placebo. Doses of efzofitimod ranged from 0.03 mg/kg up to 5.0 mg/kg. The results indicate that the drug was generally well-tolerated at all dose levels tested, with no significant adverse events or induction of anti-drug antibodies observed following efzofitimod dosing or throughout the one-month follow-up period.
The pharmacokinetics (PK) of efzofitimod following single-dose administration were linear across the evaluated dose range. Higher efzofitimod doses yielded sustained serum concentrations through the end of the one-month follow-up period that were above the predicted therapeutic threshold, supporting the potential for a once-monthly dosing regimen.
EVALUATION OF THE SAFETY, PHARMACOKINETICS AND IMMUNOGENICITY OF KRP-R120 IN HEALTHY JAPANESE VOLUNTEERS
Kyorin Pharmaceutical, our partner for development and commercialization of efzofitimod for ILD in Japan, conducted a Phase 1 clinical trial of efzofitimod, which investigated the safety, PK and immunogenicity of efzofitimod (known as KRP-R120 in Japan) in 32 healthy Japanese volunteers. In this study, efzofitimod was observed to be generally safe and well-tolerated with no drug-related serious adverse events and PK findings were consistent with previous studies of efzofitimod.